CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs

HOSPICES CIVILS DE LYO0 个研究点开始时间: 2010年7月12日最近更新: 2012年3月19日

适应症

试验速览

暂无简介。

•Patients with nephropathic cystinosis
•- Male and female subjects with confirmed diagnosis of nephropathic cystinosis (defined by clinical signs and WBC cystine level).
•- Age > 6 years.
•- Subjects receiving any oral cysteamine treatment: Cystagon? or RP103.
•- Subjects must be able to swallow their typically administered cysteamine treatment.
•- Sexually active female subjects of childbearing potential must agree to utilize the same acceptable form of contraception from day 1 through completion of the study.
•- Subjects or their parent or legal guardian must provide written informed consent prior to participation in the study.
•- Subjects covered by or having the right to social security.
•- Age and sex matched to study population :
•Age range : patients aged 6 to 11 years , patients aged 12 to 17 years, adult patients: 18 or above.
•Sex : male or female
•- Subjects or their parent or legal guardian must provide written informed consent prior to participation in the study.
•- Subjects covered by or having the right to social security.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range

•Patients with nephropathic cystinosis
•- Subjects receiving any form of cysteamine medication through a gastric tube.
•- Subjects with known hypersensitivity to cysteamine and penicillamine.
•- Females who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive urinary pregnancy test.
•- Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
•Contra-indication to MRI assessment
•Controls for metabonomic assessment
•- Any uropathology or nephropathology.

发起方

HOSPICES CIVILS DE LYO